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"Hemlibra is currently the only approved medicine for people with hemophilia A with or without factor VIII inhibitors that can be self-administered subcutaneously once weekly, every 2 weeks, or every 4 weeks," Dr. Juliana Biondo explains. ow.ly/hsXO50P28Oo #hemophilia #FDA

The American Indian/Alaska Native population showed the largest increase in maternal mortality ratios rising from 14-49.2, while the Black population consistently had the highest median state ratios, increasing from 26.7-55.4 ow.ly/V7t950P2O7o #FetalMedicine #Womenshealth

Psychological factors also come into play, as patients may have varying appetites for risk and preferences for treatment regimens, Dr. Guy Young explains in an interview with Giuliana Grossi ow.ly/721P50P6Xl9 Guy Young Children's Hospital LA #hemophilia #genetherapy #prophylaxis

Efanesoctocog alfa demonstrates promising results in new phase 3 data emphasizing the drug's potential to address unmet needs within the hemophilia community. ow.ly/FipW50P6YTk #ISTH2023 ISTH #raredisease

Individuals seeking contraception will be able to access norgestrel without consulting a health care provider, reducing barriers to contraception access. ow.ly/ra7950PaEzC #FDA #birthcontrol

The latest feature in The Unreached series delves into integrative care with expert perspectives from Guy Young, Ti-Lo, and more. Read here: ow.ly/pOFQ50Pc49O Texas Children's Children's Hospital LA Nemours Children's Health SCDAA Giuliana Grossi #integrativemedicine #chornicdisease

A year of research emphasizes the significance of collaboration, standardized approaches, and targeted therapies in rare pulmonary diseases. ow.ly/qIBr50Pmehl

A #RareDisease win last night: The FDA has approved nirogacestat tablets (Ogsiveo) to treat progressing, painful desmoid tumors. Learn more about the systemic drug here: hcplive.com/view/fda-appro… HCPLive $SWTX

Today is #RareDiseaseDay2024! All day we are highlighting the most popular coverage this year from HCPLive! To kick off the day, here's a clip where Ahmad Masri calls attention to the need for reform in developing therapies for #RareDisease. hcplive.com/view/experts-p…

In the last 12 months, the management of #IgANephropathy has undergone seismic change, with the first agent (oral budesonide; Tarpeyo) receiving full approval in late 2023. Learn more about the space and ongoing development in our #IgAN resource center: hcplive.com/clinical/berge…

In January, Alkeus Pharma announced positive interim data from the TEASE-3 clinical trial showing gildeuretinol slows the progression of Stargardt Disease, a rare ophthalmic disorder, for up to 6 years. hcplive.com/view/michael-g… #RareDiseaseDay

Algaille syndrome represents a difficult-to-treat #RareDisease in hepatology. In late 2023, data from the odevixibat program suggests the IBAT inhibitor could provide significant benefit for these patients. hcplive.com/view/odevixiba… #RareDiseaseDay2024

From October 2023: The US FDA approved an sNDA for vosoritide (VOXZOGO) to increase linear growth in pediatric patients ≤5 years of age with achondroplasia. hcplive.com/view/fda-appro… #RareDiseaseDay2024 #RareDiseaseDay

From #ASH2023: Phase 3 data show etranacogene dezaparvovec (EtranaDez), a gene therapy from Hemgenix, significantly reduced annualized bleeding rates and the number of total bleeds in hemophilia B. hcplive.com/view/etranacog… #RareDiseaseDay #RareDiseaseDay2024 CGTLive®

From our This Year in Medicine series: Dr. Robert M. Califf joins HCPLive to share perspectives on many topics, including gene and cell therapy development for rare diseases. Learn more: hcplive.com/view/reviewing… #RareDiseaseDay #RareDiseaseDay2024

In this interview: Safer, a patient with primary sclerosing cholangitis, discusses how symptoms and quality of life often go unaddressed in clinician-patient interactions. hcplive.com/view/what-clin… #RareDiseaseDay #RareDiseaseDay2024

Gina Choi, MD, A hepatologist from UCLA, breaks down the unmet need and potential for improvement within the management of autoimmune hepatitis. hcplive.com/view/autoimmun… #RareDiseaseDay #RareDiseaseDay2024

In August 2023: The US FDA approved pozelimab-bbfg (Veopoz) for the treatment of adult and pediatric patients 1 year of age and older with CHAPLE disease—the first therapy to receive such an indication. hcplive.com/view/fda-appro… #RareDiseaseDay #RareDisea

That is it from us for #RareDiseaseDay2024! Thank you to all those who acknowledged the day and an even bigger thank you to all those pushing the field into the future! hcplive.com/clinical/rare-…

Big news in #RareDisease this morning! The US FDA has approved mavorixafor (XOLREMDI; X4 Pharmaceuticals) as the first agent with an indication for patients with WHIM syndrome 12 years or older based on data from the 4WHIM trial. Learn more on HCPLive: hcplive.com/view/fda-appro…